CMTA Headlines:


CMTA Announces $1 Million in Funding for STAR Research Projects

The CMTA is awarding $1 million in research grants to industry partners and top CMT scientists worldwide, bringing to more than 50 the number of active research projects currently being funded. Every grant will have an impact on therapy development, from improving understanding of disease mechanisms to discovering new therapeutic targets and moving these into clinical trials.
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The Spring 2019 CMTA Report

The Spring 2019 CMTA Report

Clinical Trials: A Primer! In addition to our regular features, read about “A Clinical Trial for Caroline,” fast tracking and more.

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2019 CMTA Logo

CMTA Unveils New Logo

“Through STAR and our incredibly generous and engaged members, encouraging advancements have been made in the development of treatments for CMT,” said Amy Gray, CEO of the CMTA. “Our new logo represents our promise of even more breakthroughs at an accelerated pace. We will not rest until we put an end to CMT.” Read more …

Patients as Partners in Research

Patients as Partners in Research: New Opportunities to Participate

The goal of Patients as Partners in Research is to advance the work of our Strategy to Accelerate Research (STAR) and enroll the patient community in the critical work of furthering the development of treatments and ultimately a cure for CMT. Read more…

CMT1 Gene Therapy

Type 1 CMT Gene Therapy Project Now Underway

CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to Deliver Gene Replacement Therapy in X-linked Charcot-Marie-Tooth Disease.

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Research Identifies New Types of CMT

Research Project Identifies New Types of CMT

Dr. Stephan Züchner at the University of Miami is spearheading an ambitious project to identify new disease-causing mutations in patients.

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STAR Gene Therapy

The CMTA Is Accelerating Research Through Gene Therapy

The CMTA looks forward to a time when doctors are able to use genetic therapies to treat the root cause of CMT rather than prescribing medications or recommending surgery. We are already envisioning the possibilities that gene therapy holds for our community of 2.8 million people worldwide living with CMT. Learn more here …

Gilles Bouchard

STAR: The Webinar

CMTA board chairman Gilles Bouchard shares an overview of the amazing treatment-focused progress the CMTA has made over the past 10 years while highlighting STAR’s current breadth of projects, breakthroughs, and partnerships. Listen in to learn how the CMTA is using cutting-edge technologies, including stem cells, CRISPR, gene editing, gene therapy, biomarkers, high-throughput screening and axon regeneration to accelerate CMT research. View the recorded webinar at

Missy Warfield

The Warfield Family Makes a Difference

Do you stumble and fall? Do you have very high arches? Are your hands and ankles weak? For those of us with Charcot-Marie-Tooth (CMT), these questions represent our reality. And, for the Warfield family, this reality means three generations of family members enduring CMT. Read their inspiring story!

2017 CMTA Annual Report

Progress through Partnerships – Annual Report 2017

The CMTA is pleased to announce the publication of our 2017 Annual Report “Progress Through Partnerships”. Dedicating the past decade to expediting CMT research breakthroughs, the CMTA has made great strides toward treatments and a cure by bringing together researchers, pharmaceutical and biotechnology companies, and patients. Looking back, we believe 2017 stands out as one of the most pivotal years in our journey! Please read the CMTA Annual Report 2017 here.

Regenacy and CMTA

Regenacy and CMTA Announce Strategic Partnership to Evaluate Ricolinostat, a Selective HDAC6 Inhibitor

The CMTA and Regenacy Pharmaceuticals have announced a collaboration to validate the role of HDAC6 in multiple forms of Charcot-Marie-Tooth (CMT) disease and evaluate the efficacy of ricolinostat, a selective HDAC6 inhibitor, in animal models. The CMTA’s STAR Advisory Board member David Herrmann, M.B.B.Ch., has also joined Regenacy’s Scientific Advisory Board. Learn more…

Acceleron & CMTA

CMTA Announces Strategic Partnership with Acceleron Pharma to Advance CMT Treatment Options

The CMTA announced a strategic partnership with Acceleron Pharma, a Cambridge-based biopharmaceutical company dedicated to developing medicines to treat serious and rare diseases, including CMT. The CMTA’s Strategy to Accelerate Research (STAR) connects top CMT clinicians and academic researchers with pharmaceutical partners committed to developing treatments, and ultimately, a cure for CMT. Strategic alliances with corporate partners like Acceleron support innovative drug development with a goal to rapidly deliver therapies to CMT patients. Learn more…

Breakthroughs to a Cure

Breakthroughs to a Cure

Studies in two laboratory models of CMT1A not only stopped progression of the disease, but also showed improvement of some symptoms. To deliver the first treatments for CMT1A and other types of CMT, we need funding for clinical trials and further drug development.

What is CMT?

Charcot-Marie-Tooth, or CMT, is the most commonly inherited peripheral neuropathy and affects an estimated 2.8 million people.

Find Your Local Branch

The CMTA has over 80 Branches across North America to support people with CMT, raise awareness, and fund research. Connect with your nearest Branch today!

Fundraise for Our Future

Without fundraising and community volunteers, the CMTA would not be the leading force in CMT Research that it is today. Are you ready to make an impact?

Shop Our Store

Visit our store to see some of the most fun and fashionable ways to raise awareness!

The CMTA’s STAR (Strategy to Accelerate Research)

STAR Research
The CMTA’s vision is a world where patients with CMT neuropathies have therapies available to them to manage or modify their disease. That vision is poised to become reality as STAR pushes the research forward at an ever-increasing speed.

STAR – Translational Advances

The CMTA’s research is done in teams consisting of academic labs and clinical centers sponsored by the CMTA (STAR members), working together and with alliance partners in consortium team efforts. STAR Teams are sponsored by the CMTA via a rapid process of expert review, following invitation of proposals for targeted translational efforts. All sponsorship aims to directly aid the advancement of CMT therapies.
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